REGULATORY CONSIDERATIONS FOR ADVANCED THERAPY MEDICINAL PRODUCTS (ATMPS): GLOBAL PERSPECTIVES

Kshitija Jeetesh Shinde*, Navneet Nagsen Kharpe

Advanced Therapy Medicinal Products (ATMPs), including gene therapy medicinal products, somatic cell therapy medicinal products, tissue-engineered products, and certain combined advanced therapies, represent a major transformation in modern medicine. Unlike conventional small-molecule medicines, ATMPs frequently involve living cells, genetic material, engineered tissues, or combinations of biological and device components, creating distinctive challenges for product characterization, manufacturing, quality control, clinical development, regulatory assessment, and long-term safety monitoring. Regulatory authorities worldwide have therefore developed specialized frameworks to address the unique benefit–risk characteristics of these products. The European Union has established a dedicated ATMP framework under Regulation (EC) No. 1394/2007, with centralized authorization and scientific assessment involving the European Medicines Agency and its Committee for Advanced Therapies. In the United States, cellular and gene therapy products are primarily regulated by the U.S. Food and Drug Administration through the Center for Biologics Evaluation and Research. Japan has adopted a distinctive regulatory pathway for regenerative medical products that includes conditional and time-limited approval. Other jurisdictions, including India, have progressively developed regulatory guidance for cell- and gene-based therapies. Across regions, major regulatory considerations include product classification, chemistry, manufacturing and controls, potency, preclinical evaluation, clinical trial design, manufacturing consistency, traceability, pharmacovigilance, long-term follow-up, and management of post-approval changes. Despite substantial progress, differences in terminology, classification, evidence requirements, manufacturing standards, and approval pathways continue to complicate global development. International regulatory convergence and science-based harmonization will be essential for improving patient access while maintaining product quality, safety, and efficacy.